On July 9, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, reported that the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in the country of Georgia has approved its Clinical Trial Application (CTA) to conduct its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML) (Press release, Moleculin, JUL 9, 2025, View Source [SID1234654309]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East. Additionally, the Company expects the first patient to be treated in Georgia to occur by the end of August, if not sooner.

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"Building upon our recently announced CTA approval in the European Union (EU) from the European Medicines Agency (EMA), RAMPA approval of the MIRACLE trial protocol represents another important milestone and bolsters our ongoing enrollment efforts," said Walter Klemp, Chairman and CEO of Moleculin. "We expect to add 16 additional sites in Europe and the US to MIRACLE by the end of August. This will build to more than 30 sites by year-end for Part A of the MIRACLE. Adding to this progress, our recruitment in Part A of MIRACLE has hit seven subjects treated and one in screening with just our first site in Ukraine. All of this, importantly, supports our goal to report initial data on the first 45 subjects from Part A in the second half of 2025."

Mr. Klemp continued, "This early success in recruitment underscores the capability of our clinical sites, with the first ten screened subjects within just three months of site activation, and it showcases the commitment of ARENSIA Exploratory Medicine’s research clinic in Kyiv, Ukraine (ARENSIA). We are grateful for the continued international regulatory collaboration for this trial and believe it reflects the potential demand for Annamycin and the significant unmet need for better treatment options for R/R AML patients, especially venetoclax regimen failures where the outcomes from currently available therapies are considered dismal. Our team remains focused on driving enrollment and advancing this important program forward."

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 portion for purposes of measuring its primary efficacy endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.

The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

As previously announced with regard to the EU, the clinical trial approval with EMA was granted under the condition that the Company present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion (Part B) of the study. Results will be submitted as a substantial modification to the existing approved CTA.

For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

About ARENSIA Exploratory Medicine GmbH

ARENSIA Exploratory Medicine GmbH is a German operator of proprietary hospital-based research clinics, dedicated to performing complex clinical trials with novel molecules, involving patients across numerous disease areas. ARENSIA aims to speed up patients’ access to innovative therapies by collaborating with cutting-edge pharmaceutical companies and currently operates 13 state-of-the-art research clinics across the United States of America and Eastern Europe.

For more information about ARENSIA and its clinical operations, please visit www.arensia.com.

On July 9, 2025 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines, reported financial results for the fiscal quarter ended May 31, 2025, and highlighted significant progress across its clinical programs and strategic collaborations (Press release, Nurix Therapeutics, JUL 9, 2025, View Source [SID1234654310]).

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"During our second quarter, Nurix delivered important collaboration milestones, resulting in Sanofi’s extension of its license for our STAT6 program and FDA clearance of the IND for IRAK4 degrader GS-6791/NX-0479 in collaboration with Gilead," said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. "We are now entering a transformative period as we advance bexobrutideg into pivotal studies in CLL and progress our efforts to bring degrader-based therapies to patients with autoimmune diseases and inflammation."

Recent Business Highlights

Data presented at the 30th European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA2025) and the 18th International Conference on Malignant Lymphoma (ICML-18):
At EHA (Free EHA Whitepaper)2025 and ICML-18 in June 2025, Nurix presented updated Phase 1 clinical data for bexobrutideg (NX-5948), its investigational oral, brain-penetrant degrader of Bruton’s tyrosine kinase (BTK). The data demonstrated a robust objective response rate (ORR) of 80.9% across all doses in patients with relapsed or refractory CLL, including a complete response (CR) in a high-risk patient. The responses were durable, deepened over time, and were accompanied by a favorable safety profile, with no atrial fibrillation, systemic fungal infections, or dose-limiting toxicities observed. These results support the advancement of bexobrutideg to pivotal studies in CLL and underscore its potential to address significant unmet needs in B-cell malignancies.
Sanofi extended its license for Nurix’s STAT6 program, including STAT6 development candidate NX-3911: In June 2025, Nurix announced that Sanofi exercised its option to extend its license for Nurix’s STAT6 program, including the STAT6 degrader development candidate NX-3911, triggering a $15 million payment and bringing the total received by Nurix under this collaboration to $127 million. Nurix remains eligible for an additional $465 million in development, regulatory, and commercial milestones, plus future royalties and retains the option to co-develop and co-promote the program in the United States. NX-3911 is an oral, highly selective STAT6 degrader. STAT6 is a key transcription factor within the IL-4/IL-13 signaling pathways that drive inflammation in allergic and type 2 inflammatory conditions. Targeting STAT6 for degradation represents a promising therapeutic approach, supported by extensive insights from genetic studies and clinical validation of upstream biologics (IL-4/IL-13 inhibitors) and Janus kinase (JAK) inhibitors. Unlike JAK inhibition, which can impact multiple cytokine pathways and is associated with safety concerns, STAT6 degradation offers a more precise mechanism to modulate inflammation.

FDA clearance of IND application for GS-6791/NX-0479: In April 2025, Nurix announced that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for GS-6791 (previously NX-0479), a novel, first-in-class oral degrader of IRAK4 being developed in collaboration with Gilead Sciences. GS-6791 is designed to selectively degrade IRAK4, a key signaling protein that drives inflammation in autoimmune and inflammatory diseases. The clearance of this IND represents an important milestone in Nurix’s partnership with Gilead and paved the way for the initiation of first-in-human clinical trials.

Data presented at the American Academy of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting:
In April 2025, Nurix presented positive preclinical data at the AACR (Free AACR Whitepaper) Annual Meeting highlighting its portfolio of orally available, brain-penetrant degraders targeting BTK, pan-mutant BRAF, and Aurora A kinase, key drivers of oncogenic signaling and tumor growth in cancers with central nervous system (CNS) involvement. Bexobrutideg, Nurix’s lead BTK degrader, demonstrated exceptional catalytic efficiency, with a single molecule degrading approximately 10,000 BTK copies per hour, supporting its potential to deliver deep, durable responses at low doses. Nurix’s BRAF degrader showed broad preclinical activity across all three classes of BRAF mutations, including those resistant to approved therapies, while Nurix’s Aurora A degrader demonstrated significant anti-tumor activity in models of pediatric and adult cancers.

At the AACR (Free AACR Whitepaper) Annual Meeting, Nurix also presented data highlighting the transformative potential of its DEL-AI platform, which leverages a first-in-class DEL Foundation Model trained on proprietary DNA-encoded library (DEL) data to enable rapid in silico identification of novel binders for a broad range of therapeutically relevant proteins, including targets previously considered undruggable. This innovative machine learning platform has the potential to significantly accelerate the discovery of degrader-based medicines and other small molecule therapeutics for Nurix’s internal pipeline and discovery collaborations.
European Medicines Agency (EMA) granted Orphan Drug Designation (ODD) to bexobrutideg for the treatment of lymphoplasmacytic lymphoma: In July 2025, Nurix announced that the EMA granted ODD to bexobrutideg for the treatment of lymphoplasmacytic lymphoma, of which Waldenström macroglobulinemia is the most common subtype. The EMA’s Orphan Drug Designation program grants orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 5 in 10,000 people in the European Union. This designation provides several incentives to encourage the development of treatments for rare conditions, including 10 years of market exclusivity in the EU upon approval, access to protocol assistance, eligibility for centralized marketing authorization, and significant reductions in regulatory fees.
Upcoming Program Highlights*

Bexobrutideg (NX-5948): Building on the recent positive data in CLL and WM, Nurix anticipates providing additional clinical updates for bexobrutideg and remains on track to initiate pivotal trials for bexobrutideg in CLL in the second half of 2025. To support future development of bexobrutideg in autoimmune and inflammatory diseases, Nurix has expanded a new Phase 1b cohort for patients with CLL and autoimmune hemolytic anemia and is exploring the filing of a non-malignant hematology IND for autoimmune cytopenias in 2025. More information on the ongoing Phase 1a/1b trial of bexobrutideg is available at www.clinicaltrials.gov (NCT05131022).
Zelebrudomide (NX-2127): Zelebrudomide is an orally bioavailable degrader of BTK and the cereblon neosubstrates IKZF1 (Ikaros) and IKZF3 (Aiolos) designed for the treatment of relapsed or refractory B-cell malignancies. Nurix is conducting a Phase 1a/1b clinical trial, including a Phase 1b expansion cohort focused on patients with diffuse large B-cell lymphoma and mantle cell lymphoma. Nurix is enrolling a dose escalation study within the current Phase 1a/1b trial using its new chirally controlled drug product. Future clinical updates are anticipated in the second half of 2025. Additional information on the zelebrudomide clinical trial can be accessed at www.clinicaltrials.gov (NCT04830137).
NX-1607: NX-1607 is an investigational oral inhibitor of the E3 ligase Casitas B-lineage lymphoma proto-oncogene B (CBL-B) being developed for immuno-oncology indications, including a range of solid tumor types and lymphomas. Nurix is evaluating NX-1607 in an ongoing Phase 1 trial in adults in a range of oncology indications. This study includes a thorough investigation of both dose and schedule in the Phase 1a portion. Future clinical updates are anticipated in the second half of 2025. Additional information on the NX-1607 clinical trial can be accessed at www.clinicaltrials.gov (NCT05107674).
Continued pipeline advancement of strategic collaborations with Gilead, Sanofi and Pfizer: Nurix expects to continue to achieve substantial research collaboration milestones throughout the terms of its collaborations with Gilead, Sanofi, and Pfizer.
*Expected timing of events throughout this press release is based on calendar year quarters.

Fiscal Second Quarter 2025 Financial Results

Revenue for the three months ended May 31, 2025, was $44.1 million, compared with $12.1 million for the three months ended May 31, 2024. The increase was primarily due to $30 million of license revenue from the achievement of two Sanofi license extensions and a $5 million clinical milestone achieved under Nurix’s collaboration with Gilead during the three months ended May 31, 2025.

Research and development expenses for the three months ended May 31, 2025, were $78.1 million compared with $48.9 million for the three months ended May 31, 2024. The increase was primarily related to clinical, contract manufacturing and consulting costs as Nurix continued to accelerate the enrollment of patients in the ongoing trial of bexobrutideg and prepare for the initiation of pivotal trials.

General and administrative expenses for the three months ended May 31, 2025, were $14.3 million, compared with $11.7 million for the three months ended May 31, 2024. The increase was primarily due to an increase in compensation and related personnel costs and consulting costs.

Net loss for the three months ended May 31, 2025, was $43.5 million, or ($0.52) per share, compared with $44.5 million, or ($0.71) per share, for the three months ended May 31, 2024.

Cash, cash equivalents and marketable securities was $485.8 million as of May 31, 2025, compared to $609.6 million as of November 30, 2024. Cash, cash equivalents and marketable securities as of May 31, 2025, does not include a $4.0 million milestone earned in the first fiscal quarter of 2025 and received post fiscal quarter end, and a $15.0 million license extension payment received post fiscal quarter end.

On July 9, 2025 Valerio Therapeutics reported its full year results (Press release, Valerio Therapeutics, JUL 9, 2025, View Source [SID1234654342]).

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On July 9, 2025 Actithera, a radiopharmaceutical biotech company translating medicinal chemistry insights into next-generation radioligand therapies (RLTs), reported the close of an oversubscribed $75.5 million Series A financing round (Press release, Actithera, JUL 9, 2025, View Source [SID1234654293]). The financing will support the advancement of Actithera’s lead FAP asset into clinical development in multiple indications, while also enabling the continued development of its proprietary RLT discovery platform and preclinical pipeline.

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The round was co-led by founding investor M Ventures and new lead investors Hadean Ventures, Sofinnova Partners, and 4BIO Capital, with additional participation from Bioqube Ventures, Innovestor’s Life Science Fund, Investinor, Surveyor Capital (a Citadel company), and second founding investor, Arkin Bio Ventures II.

The Company’s discovery platform combines rational drug design with radiochemistry to create novel small molecule radioligands that overcome current limitations in radiopharmaceutical development. Its three-pillar platform includes first-in-class covalent targeting strategies, designed to optimize tumor residence time, while ensuring rapid systemic clearance – improving precision, safety, and efficacy. Two additional proprietary approaches further support compound differentiation and improve tumor residence time and selectivity. This platform was validated through Actithera’s work on FAP, a high-value theranostic target known for being difficult to drug with molecules that maintain prolonged tumor residency. These efforts have resulted in a FAP-directed RLT development candidate with best-in-class potential due to its optimal pharmacokinetic profile and tumor specificity.

Dr. Andreas Goutopoulos, founder and CEO, brings over 25 years of pharmaceutical and biotech industry experience, including a track record of more than a dozen development candidates. His background includes over a decade of discovery leadership at EMD Serono, where he led medicinal chemistry. In his role as Entrepreneur-in-Residence (EIR) at M Ventures, he led the scientific efforts of and supported a number of oncology small molecule biotechs. At Actithera, he is pioneering a chemistry-driven, precision approach to RLTs by integrating novel covalent-targeting chemistries, rational drug design principles and an isotope-agnostic philosophy.

"We set out to bring structure-based and kinetics-driven thinking from small molecule drug design into the world of radiopharmaceuticals," said Dr. Andreas Goutopoulos. "This oversubscribed Series A, backed by a truly global and experienced investor syndicate, is strong validation of our approach. We engineer our radioconjugates for extended retention within tumors, making them ideally suited for longer-lived radionuclides and ultimately delivering more convenient dosing schedules and enhanced efficacy and safety for patients."

Karl Naegler, incoming Board member and Partner at Sofinnova Partners, noted: "Actithera is applying Big Pharma discipline to an emerging field with enormous potential. Its radioligand therapies represent a meaningful shift in oncology, with the opportunity to redefine the therapeutic index. We’re excited to support that vision."

Roger Franklin, incoming Board member and Partner at Hadean Ventures, added: "Actithera stands out as one of the most thoughtfully constructed radiopharma platforms we’ve seen, combining smart molecular design with a deep understanding of tumor biology and clinical need. The team’s work to align pharmacokinetics with therapeutic effect could transform how patients experience and benefit from radioligand therapies."

Therese Liechtenstein, incoming Board member and Investment Director at 4BIO Capital, added: "We are honored to support Actithera, whose molecules address key challenges in the nascent radioligand therapies space; a large therapeutic window through high tumor retention and low systemic exposure, applied to a lead program that has significant pan-tumor therapeutic potential."

Hakan Goker, current Chairman of Actithera, and Managing Director at M Ventures, said: "We are excited to see Actithera evolve from the one-person ideation we seeded with Andreas and Arkin to the transatlantic company it has become today. The innovative chemistry platform built and the first-in-class approach on FAP have the potential for a large impact in the RLT field and a significant benefit for patients. We welcome the new investor group and Board members to the company aligned with this bold vision of building the next generation of RLTs."

As part of the Series A financing, Roger Franklin, Partner at Hadean Ventures, Karl Naegler, Partner at Sofinnova Partners, Therese Liechtenstein, Investment Director at 4BIO Capital, and Debbie Dumont, Managing Partner at Bioqube Ventures will join the Actithera Board of Directors, including Noga Yerushalmi, Investment Director at M Ventures, who is currently on the Board.

On July 9, 2025 Revolution Medicines, Inc. (Nasdaq: RVMD), a late-stage clinical oncology company developing targeted therapies for patients with RAS-addicted cancers, and Iambic Therapeutics, a clinical-stage life science and technology company developing novel medicines using its AI-driven discovery and development platform, reported a technology and research collaboration to pursue novel drug candidates using Iambic’s leading AI models (Press release, Revolution Medicines, JUL 9, 2025, View Source [SID1234654312]).

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In this multi-year agreement, Iambic will use structures and molecular libraries provided by Revolution Medicines to train bespoke versions of NeuralPLexer, Iambic’s industry-leading model for protein-ligand structure prediction. Revolution Medicines will also have access to Iambic’s PropANE model, a pre-trained graph neural network deployed across dozens of drug properties for lead selection and optimization.

"We are impressed with the Iambic team and the potential of their platform to enable the discovery of novel compounds on behalf of Revolution Medicines’ portfolio," said Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines. "The capabilities of Iambic’s AI-driven discovery platform, partnered with our unique collection of proprietary data, present an opportunity to rapidly explore oncology targets known to be challenging to address through conventional drug discovery approaches."

Iambic will build custom versions of NeuralPLexer and other technologies trained on Revolution Medicines’ proprietary data to inform drug discovery against novel drug targets. Both companies will have access to the improved models, and each company retains rights to a limited number of exclusive targets as well as the ability to designate additional exclusive targets to pursue independently.

"We are thrilled to work with a visionary company like Revolution Medicines on what we believe is a novel biopharma collaboration," said Tom Miller, Ph.D., chief executive officer and co-founder of Iambic. "This collaboration enables us to expand the impact of our AI technologies as we endeavor to build industry-leading models and medicines. We have applied approaches that underly this collaboration internally and are excited to offer these approaches externally to great partners like Revolution Medicines."

Under the agreement, Iambic will receive up to $25 million through a combination of upfront and expected near-term performance-based milestone payments as well as ongoing research and development reimbursements.

About Iambic’s AI-Driven Discovery Platform
The Iambic AI-driven platform was created to address the most challenging design problems in drug discovery, leveraging technology innovations such as NeuralPLexer for best-in-class prediction of protein and protein-ligand structures. The integration of physics principles into the platform’s AI architectures improves data efficiency and allows molecular models to venture widely across the space of possible chemical structures. The platform enables identification of novel chemical modalities for engaging difficult-to-address biological targets, discovery of defined product profiles that optimize therapeutic window, and multiparameter optimization for highly differentiated development candidates. Through close integration of AI-generated molecular designs with automated chemical synthesis and experimental execution, Iambic completes design-make-test cycles on a weekly cadence.